November 2018 Newsletter
In this Issue:
Message from the Executive Director | Family Services Update
#GivingTuesday | Clinical Trial Updates | In the Spotlight
National Family Caregivers Month | Shop and Support the NNPDF
Fundraising and Awareness Events
NNPDF Equipment Exchange | Stay Connected With Us
Dear Friends and Families,
Fall is always a time of change. I am simultaneously struck by the beauty of the leaves changing while also pulled by the need to prepare for winter. It is a challenge sometimes to remember to pause and savor beautiful moments. At the office, we have gone from our Niemann-Pick Awareness Month in October to November’s National Family Caregivers Month and there has been a great deal of important information to communicate and share. Be sure that you’re receiving all our news and staying up-to-date by social media, our blog, and website updates, in addition to our newsletter.
As you remember, we launched the International Niemann-Pick Disease Registry (INPDR) recently. It’s an important tool to collect patient-reported and clinical data, and a valuable community resource. Learnings from this registry may be used for research and treatments in the future. Have you registered for yourself or your loved one yet? To learn more about the INPDR or to register in the patient portal visit nnpdf.org/inpdr.
We appreciate our industry partners for the work they are doing to advance Niemann-Pick treatment options. We are also beyond appreciative to all of our families and friends who help support us. We cannot do the vital community advocacy and support work that we do without your assistance. #GivingTuesday is coming up on November 27th. Please think of NNPDF on this day of generosity and ask other to do the same. By supporting the foundation you can make a direct impact on our loved ones in all stages of their Niemann-Pick journey through advocacy and support programs that provide services to NPD families.
As we approach Thanksgiving, remember to pause and savor beautiful moments in your lives. Share these moments with us, they inspire us and fill our team with hope.
Joslyn Crowe, MSW, MA
NNPDF Executive Director
November was a very exciting time here at the NNPDF, seeing all the local awareness efforts and learning more about you, the families in our membership, was very fulfilling. Thank you all for working hard to continue to raise NPD awareness locally and nationally. Together we are stronger!
One of the highlights of this fall for me personally was attending a patient advisory board with several parents of children and adults with NPC. I was able to absorb so much energy and information from this amazing group of parents, and their input will continue to help as I develop family service programs for you, our members.
I’m excited to announce that our first Family Services Connections Chat will take place on Thursday, November 29th at 8:00 pm ET/5:00 pm PT. The topic will be “Sharing Our Diagnostic Journey” and the group is limited to 5 people only. Don’t miss the opportunity to reserve your space in this inaugural session. To sign up please email me at firstname.lastname@example.org.
Have you completed the Family Service Connections Survey? I am so excited to offer small group video chats on topics you need! We had almost 50 people submit survey data. If you have not done so, please complete the survey to let us know how we can better support you.
Were you able to join board chair, Dr. Justin Hopkin as he presented a live webinar about the INPDR? If you missed the live webinar, please make sure to watch the recording here. Learn more about the INPDR today. Have you registered yet? Send me a quick email if you have any questions and also to let me know you began the process.
Please remember I am here to help you during all phases of the NPD journey. Let me know if you have any questions or if I can be of assistance.
Laurie J. Turner
Family Services Manager
The NNPDF will be participating in a national day of generosity, #GivingTuesday, on November 27th, 2018.
By supporting the foundation you can make an impact on our loved ones living with Niemann-Pick Disease. With your help, we’ll continue to raise funds which support programs that provide services to our NPD families. Consider making a gift online at www.nnpdf.org.
We Thank You!
Update from CTD Holdings, Inc.:
- CTD Holdings, Inc. Announces Partnership with Synteract to support the Extension Protocol for its Phase I Clinical Trial in the United States. Click here for complete announcement.
Update from Mallinckrodt Pharmaceuticals:
- Mallinckrodt VTS-270 Data Announcement. Click here for details.
The NNPDF posts new information regarding clinical trial updates as soon as it is received. Please review our Clinical Trial Update procedure for alerting you to these updates as they are made. READ MORE
Anne O’Connor Smith,
ASMD Patient and NNPDF Board Member
Tell us a bit about yourself, such as where you live and what you enjoy doing…
I am a retired software engineer living on the East Coast of Central Florida with my Ironman husband Mike, my dachshund, bengal cat and two mini Brazilian short-tailed (pocket) opossums. I enjoy visiting with my adult children and relatives, Kung Fu (modified), triathlons (former Ironman & marathoner) sailing, kayaking, boogie boarding, gardening and making wine from my homegrown tropical fruits.
When did receive your diagnosis? What led you to diagnosis?
I received my diagnosis at age 11 when my entire family had numerous tests for a week at Texas Children’s Hospital. Starting at age 6 I had stomach aches when I had to sit in a school desk all day. My doctor felt my spleen and liver were enlarged but my 2 years younger brother did not have that issue. My 9 years younger sister also had enlarged spleen and liver so our doctor realized it was genetic and referred us to Texas Children’s Hospital.
What were the first steps you took after diagnosis?
40 years ago we were told there was nothing that could be done. One medical student told me I would die by age 12; then the next day the genetic specialists said that was wrong but they had no idea what would happen to us. My mom said we could go back to Texas Children’s every year for free testing but I was too traumatized and refused.
How did you learn about the NNPDF?
When my sister was pregnant with her first baby she worried about us not knowing anything about our disease. I realized she was right and I found NNPDF on the internet and joined the former Type B Yahoo group (now we connect on Facebook).
What caused you to get involved with the NNPDF initially and what led you to become a Board member?
From the Yahoo group I learned about and participated in clinical trials. I also learned about and attended NNPDF Family Conferences where I met and continue to meet many inspirational families. I met Lisa and Jami Chavez first and was amazed at their sense of peace and purpose after the passing of their daughter Breann. Elissa Miller-Visoky first suggested my board participation and I was also inspired by Sandy Cowie’s board participation as well as the fighting spirit of all those affected by the disease who quickly became my long distance friends.
What changes have you seen in the Niemann-Pick environment over the past 5 years?
I have seen our community become much more hopeful, focused, involved, global and self-empowered. I believe this is powered by clinical trial participation and collaboration with the FDA, researchers, the NNPDF and other organizations.
What are your hopes for the future for yourself and for the Niemann-Pick community?
I hope we get treatments approved for all NPD types ASAP! Drug development through the FDA is such a long process! I also hope for all 50 states to test babies for NPD once treatments are available. Hopefully then we can get gene therapy tested and approved and not need IVs every 2 weeks! Treatments and a cure are within our reach!
We celebrate those caring for loved ones with Niemann-Pick disease. We know it is not always easy and we value you and the important role you play for your family.
Some things to remember*:
- You cannot be perfect
- You have a right to all of your emotions
- Depression is the most common emotion of long-term caregivers
- Set realistic expectations – for yourself and your loved one
- Learn about the disease and what you can expect
- Learn the skills you need to care for the care receiver and which ones you are or are not able to perform
- Learn to say “no” to things you cannot do
- Learn to accept help from others
- Build resilience
- Identify your button-pushers/stressors
- Identify your coping skills
Most importantly, remember that taking care of yourself is as important as taking care of someone else.
The NNPDF has resources and information available to help you throughout your Niemann-Pick journey.
*Family Caregiver Alliance: https://goo.gl/6GX8B9
Support the NNPDF while you shop this holiday season! AmazonSmile is a simple and automatic way for you to support the NNPDF every time you shop, at no cost to you. Click here for more information!
Contributions through fundraising by NNPDF members, families and friends are used to provide services and information to individuals and families affected by NPD, as well as supporting research into finding treatments. Please continue to host and support NPD fundraisers. Awareness Events promote awareness to the general public about Niemann-Pick Disease. Click here to view current and past fundraising and awareness events.
THANK YOU to those of you who have already hosted a Facebook Fundraiser! Are you interested in hosting? It is a great opportunity to raise awareness and funds for the NNPDF. If you are hosting a Facebook fundraiser please email the NNPDF Central Office at email@example.com to let us know.
Have you hosted a fundraiser for NNPDF recently? Send us your photos and we’ll share them and details from your event in upcoming newsletters!
The NNPDF Equipment Exchange program assists those who wish to donate pre-owned equipment to others who may be in need. Click here to check out all items available. If you have any questions or an interest in any items, please contact the NNPDF at firstname.lastname@example.org.
A quick reminder that if you are not receiving our NNPDF monthly newsletter via email or have recently changed your address or email, please click here to join us or to update your information.